Summary

Location
at UCLA
Dates
study started
completion around
Principal Investigator
by Perry Shieh, MD, PhD (ucla)

Description

Summary

The primary goal of this proposal is to collect motor and functional outcomes specific to FSHD over time. By collecting measures specific to FSHD, this will help ensure the best level of clinical care is being provided. Also, the hope is to speed up drug development by gaining a better understanding of how having FSHD impacts motor function and other health outcomes (i.e. breathing, wheelchair use, etc.) and how big a change in motor function would be clinically meaningful to those with FSHD.

Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD) will have approximately 450 FSHD participants followed for a minimum of 3 years. A subset of MOVE FSHD participants, approximately 200, will participate in the MOVE+ sub-study which includes whole body MRI and muscle biopsy.

Keywords

FSHD, Facioscapulohumeral Muscular Dystrophy, MOVE FSHD Study Visits

Eligibility

You can join if…

- Genetically confirmed FSHD (types 1 or 2) or clinical diagnosis of FSHD with characteristic findings on exam and an affected parent or offspring.

You CAN'T join if...

  • Unwilling or unable to provide informed consent.
  • Any other medical condition which in the opinion of the investigator would interfere with study participation.

Locations

  • David Geffen School of Medicine at UCLA accepting new patients
    Los Angeles California 90095 United States
  • Neuromuscular Disorders Program at Stanford University School of Medicine accepting new patients
    Stanford California 94306 United States

Lead Scientist at University of California Health

  • Perry Shieh, MD, PhD (ucla)
    HS Clinical Professor, Neurology, Medicine. Authored (or co-authored) 87 research publications

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
University of Kansas Medical Center
ID
NCT04635891
Study Type
Observational
Participants
Expecting 450 study participants
Last Updated